orphan drugs understanding the rare disease market and its dynamics woodhead publishing series in biomedicine

Download Book Orphan Drugs Understanding The Rare Disease Market And Its Dynamics Woodhead Publishing Series In Biomedicine in PDF format. You can Read Online Orphan Drugs Understanding The Rare Disease Market And Its Dynamics Woodhead Publishing Series In Biomedicine here in PDF, EPUB, Mobi or Docx formats.

Orphan Drugs

Author : Elizabeth Hernberg-Ståhl
ISBN : 9781908818393
Genre : Medical
File Size : 79. 10 MB
Format : PDF, Docs
Download : 692
Read : 601

Download Now


This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe

A Practical Guide To Rational Drug Design

Author : Sun Hongmao
ISBN : 9780081001059
Genre : Medical
File Size : 21. 16 MB
Format : PDF, ePub, Docs
Download : 965
Read : 396

Download Now


This book is not going to be an exhaustive survey covering all aspects of rational drug design. Instead, it is going to provide critical know-how through real-world examples. Relevant case studies will be presented and analyzed to illustrate the following: how to optimize a lead compound whether one has high or low levels of structural information; how to derive hits from competitors’ active compounds or from natural ligands of the targets; how to springboard from competitors’ SAR knowledge in lead optimization; how to design a ligand to interfere with protein-protein interactions by correctly examining the PPI interface; how to circumvent IP blockage using data mining; how to construct and fully utilize a knowledge-based molecular descriptor system; how to build a reliable QSAR model by focusing on data quality and proper selection of molecular descriptors and statistical approaches. A Practical Guide to Rational Drug Design focuses on computational drug design, with only basic coverage of biology and chemistry issues, such as assay design, target validation and synthetic routes. Discusses various tactics applicable to daily drug design Readers can download the materials used in the book, including structures, scripts, raw data, protocols, and codes, making this book suitable resource for short courses or workshops Offers a unique viewpoint on drug discovery research due to the author’s cross-discipline education background Explores the author’s rich experiences in both pharmaceutical and academic settings

Therapeutic Risk Management Of Medicines

Author : Stephen J. Mayall
ISBN : 9781908818270
Genre : Medical
File Size : 31. 32 MB
Format : PDF, ePub, Docs
Download : 716
Read : 1317

Download Now


Therapeutic risk management of medicines is an authoritative and practical guide on developing, implementing and evaluating risk management plans for medicines globally. It explains how to assess risks and benefit-risk balance, design and roll out risk minimisation and pharmacovigilance activities, and interact effectively with key stakeholders. A more systematic approach for managing the risks of medicines arose following a number of high-profile drug safety incidents and a need for better access to effective but potentially risky treatments. Regulatory requirements have evolved rapidly over the past decade. Risk management plans (RMPs) are mandatory for new medicinal products in the EU and a Risk Evaluation and Mitigation Strategy (REMS) is needed for certain drugs in the US. This book is an easy-to-read resource that complements current regulatory guidance, by exploring key areas and practical implications in greater detail. It is structured into chapters encompassing a background to therapeutic risk management, strategies for developing RMPs, implementation of RMPs, and the continuing evolution of the risk management field.The topic is of critical importance not only to the pharmaceutical and biotechnology industries, but also regulators and healthcare policymakers.Some chapters feature contributions from selected industry experts. An up-to-date practical guide on conceiving, designing, and implementing global therapeutic risk management plans for medicines A number of useful frameworks are presented which add impact to RMPs (Risk Management Plans), together with regional specific information (European Union, United States, and Japan) A comprehensive guide for performing risk management more effectively throughout a product’s life-cycle

A Biotech Manager S Handbook

Author : M O'Neill
ISBN : 9781908818157
Genre : Science
File Size : 20. 19 MB
Format : PDF, Docs
Download : 515
Read : 955

Download Now


A biotech manager's handbook lays out - in a simple, straightforward manner - for the manager or would-be entrepreneur the basic principles of running a biotech company. Most managers in biotechnology companies are working in their first company or in their first managerial role. Their expertise and experience in the scientific part of the work can be taken as a given but there is a whole range of other skills to be learned and areas of expertise to come to terms with. Small companies do not have big budgets to hire people or time to become an expert in so many areas. The book starts by outlining the state of the biopharmaceutical industry and goes on to explain the importance of planning (no matter what the size of the company). Succeeding chapters deal with the basics of intellectual property, perspectives from a university technology transfer office and how to raise some initial funding from an investor and entrepreneur. No other 'how to' manual exists for this sector Written by a range of expert professionals in each area, all in one book Is the only 'bench to bedside' book covering the whole spectrum of development

Commercializing The Stem Cell Sciences

Author : Olivia Harvey
ISBN : 9781908818140
Genre : Science
File Size : 78. 30 MB
Format : PDF
Download : 837
Read : 292

Download Now


Promising new developments in biomedical technology such as stem cell science are widely endorsed by governments keen to reduce spiralling healthcare costs, clinicians focused on patient care, and patients demanding revolutionary new treatments. Commercializing the stem cell sciences offers a comparative analysis of the commercial methods adopted in the global stem cell industries. It seeks to establish whether there is an optimum commercial model and to examine what emerging companies can learn from their predecessors. Following an introduction to stem cell sciences and the problems involved in their commercialization, the book begins with a discussion of stem cell treatments from a global perspective, and the role of innovation in the commercialization of biotechnology in general. In the second half of the book, chapters focus on the different strategies that can be employed and their relative risks and values, before a conclusion that looks at potential new developments in the field. In-depth discussion of case studies of products undergoing development Focus on commercial optimization of stem cell treatments Analysis in a global context and covering a diverse range of countries

Sterility Sterilisation And Sterility Assurance For Pharmaceuticals

Author : Tim Sandle
ISBN : 9781908818638
Genre : Medical
File Size : 77. 99 MB
Format : PDF, Mobi
Download : 993
Read : 1279

Download Now


Failure to adequately control any microbial challenge associated within process or product by robust sterilisation will result in a contaminated marketed product, with potential harm to the patient. Sterilisation is therefore of great importance to healthcare and the manufacturers of medical devices and pharmaceuticals. Sterility, sterilisation and sterility assurance for pharmaceuticals examines different means of rendering a product sterile by providing an overview of sterilisation methods including heat, radiation and filtration. The book outlines and discusses sterilisation technology and the biopharmaceutical manufacturing process, including aseptic filling, as well as aspects of the design of containers and packaging, as well as addressing the cleanroom environments in which products are prepared. Consisting of 18 chapters, the book comprehensively covers sterility, sterilisation and microorganisms; pyrogenicity and bacterial endotoxins; regulatory requirements and good manufacturing practices; and gamma radiation. Later chapters discuss e-beam; dry heat sterilisation; steam sterilisation; sterilisation by gas; vapour sterilisation; and sterile filtration, before final chapters analyse depyrogenation; cleanrooms; aseptic processing; media simulation; biological indicators; sterility testing; auditing; and new sterilisation techniques. Covers the main sterilisation methods of physical removal, physical alteration and inactivation Includes discussion of medical devices, aseptically filled products and terminally sterilised products Describes bacterial, pyrogenic, and endotoxin risks to devices and products

Working With Ferns

Author : Helena Fernández
ISBN : 1441971629
Genre : Science
File Size : 38. 15 MB
Format : PDF, ePub
Download : 540
Read : 887

Download Now


This well timed volume features a selection of chapters composed by experts in their respective fields. It covers a broad range of topics, from its fundamental biology to the fern’s population genetics and environmental and therapeutic applications.

Microfluidic Devices For Biomedical Applications

Author : Xiujun James Li
ISBN : 9780857097040
Genre : Technology & Engineering
File Size : 31. 18 MB
Format : PDF, ePub
Download : 425
Read : 821

Download Now


Microfluidics or lab-on-a-chip (LOC) is an important technology suitable for numerous applications from drug delivery to tissue engineering. Microfluidic devices for biomedical applications discusses the fundamentals of microfluidics and explores in detail a wide range of medical applications. The first part of the book reviews the fundamentals of microfluidic technologies for biomedical applications with chapters focussing on the materials and methods for microfabrication, microfluidic actuation mechanisms and digital microfluidic technologies. Chapters in part two examine applications in drug discovery and controlled-delivery including micro needles. Part three considers applications of microfluidic devices in cellular analysis and manipulation, tissue engineering and their role in developing tissue scaffolds and stem cell engineering. The final part of the book covers the applications of microfluidic devices in diagnostic sensing, including genetic analysis, low-cost bioassays, viral detection, and radio chemical synthesis. Microfluidic devices for biomedical applications is an essential reference for medical device manufacturers, scientists and researchers concerned with microfluidics in the field of biomedical applications and life-science industries. Discusses the fundamentals of microfluidics or lab-on-a-chip (LOC) and explores in detail a wide range of medical applications Considers materials and methods for microfabrication, microfluidic actuation mechanisms and digital microfluidic technologies Considers applications of microfluidic devices in cellular analysis and manipulation, tissue engineering and their role in developing tissue scaffolds and stem cell engineering

Natural Antimicrobial Agents

Author : Jean-Michel Mérillon
ISBN : 9783319670454
Genre : Science
File Size : 30. 49 MB
Format : PDF, Mobi
Download : 848
Read : 519

Download Now


Documenting the latest research in the field of different pathogenic organisms, this book presents the current scenario about promising antimicrobials in the following areas: Part I. Plants as source of antibacterials, Part II. Naturally occurring antifungal natural products, Part III. Antiparasitic natural products, Part IV. Antiviral natural products. Renowned scientists from the globe have been selected as authors to contribute chapters. Use of plants for various ailments is as old as human civilization and continuous efforts are being made to improve medicinal plants or to product their bioactive secondary metabolites in high amounts through various technologies. About 200,000 natural products of plant origin are known and many more are being identified from higher plants and micro-organisms. Some plants based drugs are used since centuries and there is no alternative medicine for many such drugs as cardiac glycosides. Drug discovery from medicinal plants or marine micro-organisms continues to provide an important source of new drug leads. Research on new antibacterials represents a real and timely challenge of this century, particularly for the treatment of infections caused by clinical isolates that show multidrug resistance. The main microorganisms involved in the resistance process have been identified and given the acronym ESKAPE for Enterococcus faecium, Staphylococcus aureus, Klebsiella pneumoniae, Acinetobacter baumanii, Pseudomonas aeruginosa and Enterobacteriaceae. Multidrug resistant Mycobacterium tuberculosis including highly drug-resistant strains (XDR-TB) has also emerged as one of the most important clinical challenges of this century. Plants of diverse taxa and marine micro-organisms are rich source of these antimicrobials. An attempt has been made to compile the recent information about natural sources of antibacterials and their sustainable utilization. Increased panic of these pathogens warrants a growing demand for research to undertake the threat of multidrug resistance. The search for new antifungal, antiparasitic and antiviral natural products is far from devoid of interest. According to the WHO report in 2013, malaria still represents some 207 million cases worldwide and more than 3 billion of people are still exposed to this risk. Similarly, about 350 million people are considered at risk of contracting leishmaniasis. The fight against some viruses also requires that the research on natural products continue. For example, even if an antiretroviral with direct action was recently approved in Europe in 2013, its high cost does not allow to offer it to an exposed population in countries where the cost of drugs remains a problem for a large part of the population. These books are useful to researchers and students in microbiology, biotechnology, pharmacology, chemistry and biology as well as medical professionals.

Biobetters

Author : Amy Rosenberg
ISBN : 9781493925438
Genre : Medical
File Size : 43. 61 MB
Format : PDF, ePub
Download : 472
Read : 833

Download Now


“Biobetters: Protein Engineering to Approach the Curative” discusses the optimization of protein therapeutic products for treatment of human diseases. It is based on the fact that though numerous important therapeutic protein products have been developed for life threatening and chronic diseases that possess acceptable safety and efficacy profiles, these products have generally not been reexamined and modified for an improved clinical performance, with enhancements both to safety and efficacy profiles. Advances in protein engineering, coupled with greatly enhanced understanding of critical product quality attributes for efficacy and safety, make it possible to optimize predecessor products for clinical performance, thereby enhancing patient quality of life and with the potential for great savings in health care costs. Yet despite such knowledge, there is little movement towards such modifications. This book examines engineering protein therapeutic products such that they exhibit an optimal, not just an adequate, clinical performance profile. Two product classes, therapeutic enzymes for lysosomal storage diseases (enzyme replacement therapies, ERT) and monoclonal antibodies (mAbs), are used as examples of what modifications to such proteins could be made to enhance clinical performance, “closer to a cure” as it were. For ERT, the key to optimizing clinical performance is to ensure the ERT is endowed with moieties that target the protein to the relevant target tissue. Thus, for Gaucher Disease, our best example of how to optimize an ERT to address a disease that manifests in specific target tissues (macrophages and monocytes), the enzyme has been extensively modified to target macrophages. For diseases such as Pompe Disease, largely a disorder of muscle, optimal performance of ERT will depend on endowing the enzyme with the ability to be taken up via the Mannose 6 Phosphate Receptor, and so one of the chapters in the book will discuss such approaches. Moreover, a major failure of biotechnology based products is to gain access to the CNS, a key target tissue in numerous diseases. Thus, a chapter has been devoted to strategies to access the CNS. Additionally, immune responses to therapeutic proteins can be highly problematic, eliminating the efficacy of life saving or highly effective protein therapeutics. This is especially poignant in the case of Pompe Disease wherein great improvement in muscle strength and functionality is lost following development of an immune response to the ERT with consequent patient deterioration and death. Thus, a chapter regarding protein engineering, as well as other non-clinical approaches to diminishing immunogenicity is a valuable part of the book. Monoclonal antibodies (mAbs) can be engineered to bind targets relevant to a wide variety of diseases; binding affinity, however, is only part of the equation and one of the chapters will present a molecular assessment approach that balances affinity with pharmacokinetics and manufacturability. As with other proteins immunogenicity can be problematic, being responsible for loss of efficacy of anti-TNF mAbs, often after prolonged successful treatment. The authors will also share their perspective on the consequences of physico-chemical modifications occurring to mAbs once they reach the circulation or their target, a research area open to further development from a protein engineering as well as analytical perspective. This book will also discuss novel platforms for protein therapeutics, technologies that exceed mAbs with respect to potency, and hence, potentially efficacy. These platforms consist largely of repeat domain proteins with very high affinity for their target ligands, but while potentially more efficacious, immunogenicity may be a major problem limiting use. The economics surrounding the issue of biobetters is another high-profile issue - this final chapter will explore the incentives and disincentives for developing biobetters and consider incentives that might make their pursuit more rewarding.

Top Download:

Best Books